Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment

Last updated: April 3, 2024
Sponsor: Takeda
Overall Status: Active - Recruiting

Phase

2/3

Condition

Dementia

Mild Cognitive Impairment

Memory Problems

Treatment

Elaprase

Idursulfase-IT

Clinical Study ID

NCT06031259
TAK-609-3001
2023-504127-90
  • Ages 3-19
  • Male

Study Summary

The study is an extension of two previous studies (HGT-HIT-046 [NCT01506141] and SHP609-302 [NCT02412787]). Participants must have completed one of the previous studies. The main aim of this study is to collect more information about the safety of the treatments, idursulfase-IT and elaprase, in children and adults with Hunter syndrome and cognitive impairment. Participants will receive the same treatment as in the previous studies.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. The participant must have completed end of study assessments in studies HGT-HIT-046 [NCT01506141] or SHP609-302 [NCT02412787] and received a clinical benefit fromidursulfase-IT in the opinion of the investigator.
  2. The participant, or participant's legally designated representative, must have beeninformed of the nature of this open-label extension and must have voluntarily signedan Institutional Review Board (IRB)/Independent Ethics Committee (IEC) approvedinformed consent form after all relevant aspects of the study have been explained anddiscussed. Written consent of the participant's legally designated representative (ifapplicable) and the participant's consent/assent, as relevant, must be obtained.
  3. The participant has continued to receive elaprase on a regular basis in studiesHGT-HIT-046 or SHP609-302.

Exclusion

Exclusion Criteria:

  1. The participant has experienced, in the opinion of the investigator, a safety ormedical issue that contraindicates treatment with idursulfase-IT, including, but notlimited to, uncontrolled seizure disorder, bleeding disorder, and clinically relevanthypertension.
  2. The participant has clinically relevant intracranial hypertension.
  3. The participant is enrolled in another clinical study, other than studies HGT-HIT-046or SHP609-302, that involves clinical investigations or use of any investigationalproduct (drug or [intrathecal/spinal] device) within 30 days prior to study enrolmentor at any time during the study.

Study Design

Total Participants: 8
Treatment Group(s): 2
Primary Treatment: Elaprase
Phase: 2/3
Study Start date:
March 05, 2024
Estimated Completion Date:
June 01, 2034

Study Description

The drug being tested in this study is called idursulfase. Idursulfase is being tested for long term safety in participants with Hunter syndrome and cognitive impairment.

The study will enroll up to approximately 8 patients. Participants will receive idursulfase-IT via intrathecal drug delivery device (IDDD) once monthly along with elaprase intravenous (IV) infusion, weekly.

This multi-center trial will be conducted in France and Canada. The overall time to participate in this study is approximately 10.4 years.

Connect with a study center

  • M.A.G.I.C. Clinic

    Calgary, Alberta T2E 7Z4
    Canada

    Active - Recruiting

  • M.A.G.I.C. Clinic LTD

    Calgary, Alberta T2E 7Z4
    Canada

    Site Not Available

  • University of British Columbia

    Vancouver, British Columbia V6T1Z3
    Canada

    Site Not Available

  • Hospital for Sick Children

    Toronto, Ontario M5G1X8
    Canada

    Site Not Available

  • The Hospital for Sick Children

    Toronto, Ontario M5G1X8
    Canada

    Site Not Available

  • Hopital Femme Mere Enfant

    Bron, Auvergne-Rhone-Alpes 02 69229
    France

    Site Not Available

  • Hospices Civils de Lyon

    Lyon Cedex, 69229
    France

    Site Not Available

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